Phase II JCV infection trial approved

Inhibikase Therapeutics has received clearance to undertake a phase II trial of IkT-001 against JCV infection, which can cause PML in immunodeficient patients

Inhibikase Therapeutics has received clearance from the US Food and Drug Administration (FDA) to begin a phase II proof-of-concept trial of its lead compound IkT-001, a host-directed kinase inhibitor, against John Cunningham human polyomavirus (JCV) infection.

JCV infection, which is very common in the general populace, is a causative agent of Progressive Multifocal Leukoencephalopathy (PML) in patients with chronic or drug-induced immunodeficiency. In these patients, PML is fatal in more than 50 per cent of patients, as the JCV virus creates lytic infection of the brain, interrupting cognitive and motor function. In pre-clinical models, however, IkT-001 was shown to be able to block JCV viral entry into host cells, suppressing the development of PML.

The phase II trial will be a four-week, open-label, multicentre, sequential dose-ranging study of the safety and pharmacokinetic profile of IkT-001 against JCV in 48 at-risk patients: those with relapsing multiple sclerosis (MS) who receive Tysabri (natalizumab) infusion therapy. The study will also examine the compound's antiviral effect as measured by the urinary excretion of JCV.

PML currently has no approved treatment, meaning that IkT-001 will be eligible for orphan drug status if it continues to progress through trials to market. Additionally, as IkT-001 utilises a common mechanism of action that enables treatment for bacterial and viral infectious disease, an efficacious outcome in this phase II study could clear a path for multiple indications using the same pharmaceutical approach, according to Inhibikase.

In the last year, both David Gryska, former chief financial officer of Celgene, and Dr Steven Gilman, executive vice president and chief scientific officer of Cubist Pharmaceuticals, have joined the board of directors of Inhibikase, an Atlanta, Georgia-based private biopharmaceutical company that began operations just 23 months ago. The company aims to cut development time to proof-of-concept by more than half, potentially bringing products to market in as little as five years from initiation of R&D.

Using this speedy approach, Inhibikase projects IkT-001 will be accelerated into the clinic at a late trial stage by the close of the year for both PML and polyomavirus-associated nephropathy (PVAN). A second-generation compound, IkT-014, which has four-fold greater activity and is believed to function through the same mechanism of action, is already in pre-clinical development.